Advancements In CRISPR-CAS9: A New Era for Genome Editing in Gene Therapy and Stem Cell Applications
Keywords:
CRISPR-Cas9, Gene Therapy, Stem Cell Research, Ethical Implications, Genome Editing, Public Perception, Genetic Disorders, Ethical GovernanceAbstract
Purpose: This study explores the advancements in CRISPR-Cas9 technology and its potential to revolutionize gene therapy and stem cell applications. It aims to assess the level of public awareness, the perceptions surrounding its effectiveness in treating genetic disorders, ethical implications, and its promise in stem cell research.
Aim: The primary objective of this paper is to evaluate the role of CRISPR-Cas9 in gene therapy for genetic disorders and its application in advancing stem cell therapies. Additionally, the study seeks to identify the ethical concerns and future outlook of CRISPR-Cas9 from a diverse group of participants with varying levels of expertise and familiarity with the technology.
Approach: A mixed-methods survey was employed to collect both quantitative and qualitative data from 130 participants, including researchers, healthcare professionals, and the general public. The questionnaire consisted of sections covering demographic data, awareness of CRISPR-Cas9, its potential in gene therapy, ethical considerations, and its role in stem cell research.
Methods: The survey included Likert-scale questions to quantify participant attitudes and opinions on the effectiveness, ethical concerns, and future of CRISPR-Cas9, alongside open-ended questions for qualitative feedback. Data was analyzed using descriptive statistics for quantitative data and thematic analysis for qualitative responses.
Results: The findings revealed strong confidence in CRISPR-Cas9's potential to treat genetic disorders, with over 70% of participants expressing optimism about its effectiveness. While a significant proportion of participants showed support for CRISPR-Cas9’s role in stem cell therapies, there were notable ethical concerns, particularly regarding its use in human gene editing and "designer babies."
Findings: The study identified key areas of public interest, including the effectiveness of CRISPR-Cas9 in genetic disease treatment, its ethical implications, and its transformative potential for regenerative medicine and stem cell applications. Despite these positive responses, concerns about the regulation and ethical governance of CRISPR-Cas9 remain critical.
Theoretical Implications: This paper provides insight into the societal perception of CRISPR-Cas9, contributing to the theoretical understanding of how advanced gene editing technologies are viewed across different sectors. The findings underline the need for comprehensive regulatory frameworks to address ethical concerns and ensure the responsible application of CRISPR-Cas9 in medical treatments.
Practical Implications: The results of this study have practical implications for the future of gene therapy and stem cell research. It highlights the necessity for greater public education on CRISPR-Cas9 and the importance of developing ethical guidelines to govern its use in human genetic modification.
Originality/Value: This paper offers a comprehensive overview of public perceptions of CRISPR-Cas9, blending both scientific and societal perspectives. It contributes to the growing body of knowledge on the ethical, scientific, and societal implications of gene editing technologies and serves as a foundation for future research and policy development in this field
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References
Bolideei, M., et al., Applications of Gene Editing and Nanotechnology in Stem Cell-Based Therapies for Human Diseases. Stem Cell Reviews and Reports, 2025: p. 1-30.
Aljabali, A.A., M. El-Tanani, and M.M. Tambuwala, Principles of CRISPR-Cas9 technology: Advancements in genome editing and emerging trends in drug delivery. Journal of Drug Delivery Science and Technology, 2024. 92: p. 105338.
Patmanathan, S.N., et al., CRISPR/Cas9 in stem cell research: current application and future perspective. Current Stem Cell Research & Therapy, 2018. 13(8): p. 632-644.
Abusalah, M.A.H., E.N.S.E. Abd Rahman, and O.P. Choudhary, Evolving trends in stem cell therapy: an emerging and promising approach against various diseases. International Journal of Surgery, 2024. 110(11): p. 6862-6868.
Lotfi, M., et al., Recent advances in CRISPR/Cas9 delivery approaches for therapeutic gene editing of stem cells. Stem cell reviews and reports, 2023. 19(8): p. 2576-2596.
De Masi, C., et al., Application of CRISPR/Cas9 to human-induced pluripotent stem cells: from gene editing to drug discovery. Human genomics, 2020. 14: p. 1-12.
Tavakoli, K., et al., Applications of CRISPR-Cas9 as an advanced genome editing system in life sciences. BioTech, 2021. 10(3): p. 14.
Paolini Sguazzi, G., et al., Induced pluripotent stem cells (iPSCs) and gene therapy: A new era for the treatment of neurological diseases. International journal of molecular sciences, 2021. 22(24): p. 13674.
Hussen, B.M., et al., Revolutionizing medicine: recent developments and future prospects in stem-cell therapy. International Journal of Surgery, 2024. 110(12): p. 8002-8024.
Tao, R., et al., Revolutionizing cancer treatment: enhancing CAR-T cell therapy with CRISPR/Cas9 gene editing technology. Frontiers in Immunology, 2024. 15: p. 1354825.
Kim, E.J., K.H. Kang, and J.H. Ju, CRISPR-Cas9: a promising tool for gene editing on induced pluripotent stem cells. The Korean Journal of Internal Medicine, 2017. 32(1): p. 42.
Baumann, M., CRISPR/Cas9 genome editing–new and old ethical issues arising from a revolutionary technology. NanoEthics, 2016. 10: p. 139-159.
Zhang, M.-L., H.-B. Li, and Y. Jin, Application and perspective of CRISPR/Cas9 genome editing technology in human diseases modeling and gene therapy. Frontiers in Genetics, 2024. 15: p. 1364742.
Allemailem, K.S., et al., Innovative strategies of reprogramming immune system cells by targeting CRISPR/Cas9-based genome-editing tools: a new era of cancer management. International Journal of Nanomedicine, 2023: p. 5531-5559.
Salas-Mckee, J., et al., CRISPR/Cas9-based genome editing in the era of CAR T cell immunotherapy. Human vaccines & immunotherapeutics, 2019.
Raikwar, S.P., et al., Next generation precision medicine: CRISPR-mediated genome editing for the treatment of neurodegenerative disorders. Journal of Neuroimmune Pharmacology, 2019. 14: p. 608-641.
Ullah, I., CRISPR/Cas9-Based Genome Editing in Precision Medicine: Current Trends and Recent Advancements. Genetics, 2024. 1: p. 100001.
Koniali, L., C.W. Lederer, and M. Kleanthous, Therapy development by genome editing of hematopoietic stem cells. Cells, 2021. 10(6): p. 1492.
Mangala, K., et al., CRISPR-CAS9 GENE EDITING IN PHARMACEUTICALS: CURRENT APPLICATIONS AND FUTURE PROSPECTS. Biochemical & Cellular Archives, 2023. 23.
Bhad, V., G. Bhalerao, and R. Deokar, CRISPR-CAS 9 In Gene Editing: Innovations, Applications and Ethical Challenges. Int. J. Sci. R. Tech, 2024. 1(11).
Goel, R., CRISPR/Cas9-mediated genome editing: from basic research to gene therapy. International Journal of Research in Medical Sciences, 2024. 12(6): p. 2200.
Bhagat, M., et al., Gene Therapy: Towards a New Era of Medicine. AAPS PharmSciTech, 2024. 26(1): p. 17.
Doudna, J.A. and E. Charpentier, The new frontier of genome engineering with CRISPR-Cas9. Science, 2014. 346(6213): p. 1258096.
Ansori, A.N., et al., Application of CRISPR-Cas9 genome editing technology in various fields: A review. Narra J, 2023. 3(2): p. e184.
Arjmand, B., et al., The horizon of gene therapy in modern medicine: advances and challenges. Cell Biology and Translational Medicine, Volume 8: Stem Cells in Regenerative Medicine, 2020: p. 33-64.
Selvakumar, S.C., et al., CRISPR/Cas9 and next generation sequencing in the personalized treatment of Cancer. Molecular Cancer, 2022. 21(1): p. 83.
Zhang, H., et al., Application of the CRISPR/Cas9-based gene editing technique in basic research, diagnosis, and therapy of cancer. Molecular cancer, 2021. 20: p. 1-22.
Salsman, J. and G. Dellaire, Precision genome editing in the CRISPR era. Biochemistry and cell biology, 2017. 95(2): p. 187-201.
Wang, J.Y. and J.A. Doudna, CRISPR technology: A decade of genome editing is only the beginning. Science, 2023. 379(6629): p. eadd8643
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